Familial dysautonomia (FD) is an autosomal recessive neurodevelopmental disorder.

Complement Therapeutics GmbH has obtained clearance for CTx-001, a gene therapy candidate.

Cirrus Therapeutics Inc. has closed an $11 million seed financing to advance its pipeline of gene and cell therapies designed to preserve sight and extend ocular healthspan in patients with chronic blinding diseases.

Neuromyelitis optica spectrum disorder (NMOSD) is neuroinflammatory disease characterized by optic neuritis and myelitis with presence of anti-aquaporin-4 (AQP4) antibodies. Satralizumab is an anti-IL-6 receptor (IL-6R) humanized antibody approved for preventing relapses in NMOSD (AQP4 IgG positive) in adults and children in Japan.

Opus Genetics Inc. will be sitting down with the U.S. FDA to talk about positive three-month data from the pediatric cohort of its ongoing phase I/II trial called OPGx-LCA5-1001 – partially funded by the agency – evaluating OPGx-LCA5, a gene augmentation therapy for ultra-rare Leber congenital amaurosis type 5 (LCA5). The affliction is a severe form of retinal dystrophy that renders babies blind in the first year of life.

Iveena Delivery Systems Inc. has been awarded a $2 million phase II Small Business Innovation Research (SBIR) grant from the National Eye Institute (NEI) to advance the development of novel topical eye drops to control pediatric myopia and other refractive disorders.